StemCells, Inc. – Therapeutic Programs: Clinical Trials

We are currently conducting or have completed clinical trials of our proprietary HuCNS-SC product candidate (purified human neural stem cells) in spinal cord injury, dry age-related macular deneneration, and intwo fatal neurodegenerative disorders:

InDecember 2010, wereceived authorization from Swissmedic, the Swiss regulatory agency for therapeutic products, to initiatea Phase I/II clinical trial of our HuCNS-SC cells in chronic spinalcord injury. The trial initiated in March 2011 and iscurrently open forenrollment. The trial is beingconductedinSwitzerland at the Balgrist University Hospital, University of Zurich. Learnmore

The trial is designed to assess both safety and preliminary efficacy in patients with varying degrees of paralysis who are three to 12 months post-injury, and will progressively enroll patients based upon the severity of injury. The trial will enroll 12 patients in Europe with thoracic (chest-level) spinal cord injury, and will include both complete and incomplete injuries as classified by the American Spinal Injury Association (ASIA) Impairment Scale. The first cohort will include patients classified as ASIA A. These patients have what is considered to be a "complete" injury, or no movement or feeling below the level of the injury. The second cohort will progress to patients classified as ASIA B, or patients with some degree of feeling below the injury. The third cohort will consist of patients classified as ASIA C, or patients with some degree of movement below the injury. In addition to assessing safety, the trial will evaluate preliminary efficacy using defined clinical endpoints, such as changes in sensation, motor, and bowel/bladder function.

All patients will receive HuCNS-SC cells through direct transplantation into the spinal cord, and will be temporarily immunosuppressed. Following transplantation, the patients will be evaluated regularly over a 12-month period in order to monitor and evaluate the safety and tolerability of the HuCNS-SC cells, the surgery and the immunosuppression, and to measure any recovery of neurological function below the injury site. As we intend to follow the effects of this therapy long-term, a separate four-year observational study will be initiated at the conclusion of this trial.

For enrollment inquiries, pleasesubmit our contact us form.

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In November 2009, we initiated a Phase I trialof our HuCNS-SCcellsin PMD, a myelination disorder that primarily affects young children. The trial is being conducted at the University of California, San Francisco (UCSF) Benioff Children's Hospital. Learnmore

In December 2008 the US Food and Drug Administration (FDA) allowed our Investigational New Drug (IND) application to conduct a clinical trial of our HuCNS-SC product candidate in patients with PMD, and in November 2009 we initiated the trial at UCSF Benioff Childrens Hospital. In February 2010 our cells were used to treat the first patient enrolled in the trial, marking the first time that neural stem cells have been transplanted as a potential treatment for a myelination disorder. In February 2011,the fourth and final patient was enrolled and dosed. Results of the trial will bereported in early 2012.

This Phase I trial is designed to assess the safety and preliminary efficacy of our HuCNS-SC cells as a potential treatment for PMD. While the primary focus in this first trial is safety, we will also be looking for evidence of new myelin formation in the patients brains following the transplantation of our cells, as well as any signs of improved neurological function. We have enrolled four patients with connatal PMD, the most severe form of the disease. All patients were transplanted with the HuCNS-SC cells and are being evaluated regularly over a 12-month period in order to monitor and evaluate the safety and tolerability of the HuCNS-SC cells, the surgery and the immunosuppression. In addition, MRI examination of the brain post-transplant may enable the measurement of new myelin formation. We are planning to follow the effects of this therapy long-term, so, as with our Phase I NCL trial, a separate, four-year observational study will be initiated at the conclusion of this trial.

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StemCells, Inc. - Therapeutic Programs: Clinical Trials